An Interview With Pfizer on the Orphan Drug Industry: Part I

In Advocacy, Clinical Development, Market Access, Partnering & Investment, Regulation & Government by Matt Turner


As part of a series of interviews conducted with leading speakers of the World Orphan Drug Congress in Geneva, Terrapinn were delighted to sit down with Adam Woodrow, VP of Global Commercial Development of the Specialty Care Business Unit at Pfizer. In the first of a two part blog, the conversation delved into improving patient access, multi-stakeholder collaborations and future challenges orphan drug developers need to be aware of.

 Where do the current and future challenges lie in bringing new orphan drugs to market?

There are inherent challenges in developing therapies to treat rare diseases, as these conditions affect small groups of people who are often geographically dispersed. This makes it difficult to identify, reach and engage with these patients about clinical studies and other research efforts. Rare diseases are often debilitating and frequently affect children, making it difficult for patients and their families to engage in efforts beyond symptom management.

Furthermore, post-approval reimbursement and patient access to urgently-needed therapies remain the biggest challenges in bringing new medicines for rare diseases to market. Reimbursement policies and timelines can vary significantly by country, creating potential disparities in patient- access to lifesaving therapeutics. Pfizer is committed to partnering with stakeholders throughout the rare disease community to achieve timely reimbursement, and we are striving to develop innovative approaches that will help ensure our medicines reach the people who need them.

What are the important aspects of making a multi-stakeholder partnership successful?

There are a number of factors that are important to the success of any multi-stakeholder partnership: aligning the cultures of diverse organizations to facilitate collaboration; identifying areas of complementary expertise that add value beyond the contributions of individual participants; and establishing a framework that aligns incentives.

Pfizer is committed to seeking partnerships around technologies or products where our scale and expertise can add value to translate scientific discoveries into new medicines for patients with rare diseases.

We strive to achieve a balance between the risk associated with a new investment and the unmet need we are seeking to address, so we can match the opportunity with the appropriately structured collaboration—whether it is a joint research endeavour or a plan to co-develop a medicine with another company. Ultimately, our goal is to engage in partnerships and collaborations which create value for the rare disease community beyond what we would be able to offer alone.

What have been/will be the key game changers altering the dynamic of the orphan drug industry for developers?

Recently, the involvement of patient advocates in the development of medicines has grown significantly. This has created a more robust, global support network for people affected by these devastating illnesses. As advocates and patients become more adept at collecting and organizing data and disease information, they will increasingly be viewed as partners in the drug development process by industry, potentially allowing for investment across a greater number of illnesses and reducing the time associated with developing new therapies. As patient organizations grow increasingly sophisticated, we are also seeing greater alignment among their constituents, who are mobilizing to advocate and raise awareness more effectively than ever before.

Adam Woodrow will be presenting on The Collaboration Imperative: why cross-stakeholder partnerships are essential to advancing progress in orphan medicines development at the forthcoming World Orphan Drug Congress in Geneva.

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